OBJECTIVES : To examine costs of care for transfusion-dependent β-thalassemia (TDT) patients including red blood cell (RBC) transfusions, iron chelation therapy, and allogeneic hematopoietic stem cell transplant (allo-HSCT).

METHODS : Retrospective analysis, using the U.S. MarketScan Commercial and Medicaid Multi-State databases, to identify patients with ≥ 8 RBC transfusions in a given 12-month period (first transfusion date = index date) between 10/1/2011 and 9/30/2016, ≥ 1 medical claim with a β-thalassemia or hemoglobin E β-thalassemia diagnosis between 3 months before and 15 months after the index date, and no evidence of allo-HSCT. Demographics, annual number of RBC transfusions, and annual treatment costs were summarized per patient. Generalized linear modeling was used to identify predictors of high costs. A subset of patients with a β-thalassemia or hemoglobin E β-thalassemia diagnosis and allo-HSCT were analyzed separately.

RESULTS : A total of 244 patients met all TDT inclusion criteria. Mean age was 22.4 years; 54.1% were female; 32.8% lived in the Northeast, 25.8% in the Midwest, 25.8% in the South, and 15.6% in the West. Patients had a median of 15 transfusions annually (min-max 8-56). Mean annual per-patient costs were $127,553 (median $97,744; min-max $1,943-$1,223,683), including $29,292 transfusion and $51,974 chelation therapy costs. High cost was predicted by oral chelation therapy volume, presence of type 2 diabetes and heart failure. There were 24 β-thalassemia patients with allo-HSCT: they had mean costs for the period covering transplant admission date + 364 days of $530,557 (median $412,107; min-max $105,988 to $1,263,969).

CONCLUSIONS : Treatment with RBC transfusion and iron chelation are life-long requirements for TDT patients; thus, the cumulative costs for basic management of TDT are substantial. Furthermore, these costs represent only a portion of the total medical cost burden of TDT which also includes ongoing monitoring of iron overload, and management of comorbidities and iron overload complications.